{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,25]],"date-time":"2026-06-25T04:44:28Z","timestamp":1782362668813,"version":"3.54.5"},"reference-count":30,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2022,4,27]],"date-time":"2022-04-27T00:00:00Z","timestamp":1651017600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2022,7]]},"abstract":"Abstract<\/jats:title>Introduction<\/jats:title>Eptacog beta is a new recombinant activated human factor VII bypassing agent approved in the United States for the treatment and control of bleeding in patients with haemophilia A or B with inhibitors 12 years of age or older.<\/jats:p><\/jats:sec>Aim<\/jats:title>To prospectively assess in a phase 3 clinical trial (PERSEPT 2) eptacog beta efficacy and safety for treatment of bleeding in children <12 years of age with haemophilia A or B with inhibitors.<\/jats:p><\/jats:sec>Methods<\/jats:title>Using a randomised crossover design, subjects received initial doses of 75 or 225 \u03bcg\/kg eptacog beta followed by 75 \u03bcg\/kg dosing at predefined intervals (as determined by clinical response) to treat bleeding episodes (BEs). Treatment success criteria included a haemostasis evaluation of \u2018excellent\u2019 or \u2018good\u2019 without use of additional eptacog beta, alternative haemostatic agent or blood product, and no increase in pain following the first \u2018excellent\u2019 or \u2018good\u2019 assessment.<\/jats:p><\/jats:sec>Results<\/jats:title>Treatment success proportions in 25 subjects (1\u201311 years) who experienced 546 mild or moderate BEs were 65% in the 75 \u03bcg\/kg initial dose regimen (IDR) and 60% in the 225 \u03bcg\/kg IDR 12 h following initial eptacog beta infusion. By 24 h, the treatment success proportions were 97% for the 75 \u03bcg\/kg IDR and 98% for the 225 \u03bcg\/kg IDR. No thrombotic events, allergic reactions, neutralising antibodies or treatment\u2010related adverse events were reported.<\/jats:p><\/jats:sec>Conclusion<\/jats:title>Both 75 and 225 \u03bcg\/kg eptacog beta IDRs provided safe and effective treatment and control of bleeding in children <12 years of age.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.14563","type":"journal-article","created":{"date-parts":[[2022,4,27]],"date-time":"2022-04-27T07:52:30Z","timestamp":1651045950000},"page":"548-556","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors"],"prefix":"10.1111","volume":"28","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-2558-2089","authenticated-orcid":false,"given":"Steven W.","family":"Pipe","sequence":"first","affiliation":[{"name":"University of Michigan Ann 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